Antisense Drug Makes Sense as Potential Broad-Acting Cancer Therapy

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(ChemotherapyAdvisor) — The search for a drug that can treat many different forms of cancer may be over. This comes with news that Isis Pharmaceuticals has initiated a Phase 1 study targeting the expression in tumors of signal transducer and activator of transcription 3 (STAT3), a gene that is ubiquitous and overexpressed in many tumor types. This has led many pharmaceutical companies to pursue this drug target as a potential therapeutic against a wide range of solid and liquid tumors. However, Isis is aiming the power of their new compound, known as ISIS-STAT3Rx, at cancers with the greatest unmet medical need — hepatocellular carcinoma (HCC) and ovarian cancer.

ISIS-STAT3Rx selectively inhibits the overexpression of its target gene through an antisense mechanism, which reduces STAT3's activity in the growth, survival, metastasis, and invasiveness of tumors. Inhibition of STAT3 has been shown to block induction of tumor-associated cytokines (e.g., IL-6, IL-1, TGF-β, and IL-10), all of which are potential biomarkers in future clinical trials for the drug.

The selectivity and potency of the drug is due to the company's advanced, proprietary chemistry platform known as Generation 2.5, and may help oncologists to treat the most difficult to treat cancers. ISIS-STAT3Rx is the first drug developed using this platform.

For more information from Isis Pharmaceuticals:


Antisense Drug Discovery Platform

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